Abstract: Provided herein are gene therapy methods for the treatment of mucopolysaccharidosis type IVA (MPS IVA) involving the use of recombinant adeno-associated viruses (rAAVs) to deliver human N-acetylgalactosamine-6-sulfate sulfatase (hGALNS) to the bone of a human subject diagnosed with MPS IVA. Also provided herein are rAAVs that can be used in the gene therapy methods and methods of making such rAAVs.

Abstract: A pharmaceutical composition for treatment of a tumor expressing at least one of a plurality of receptor proteins including CD24, Axl, DC-SIGN, Tyro3, MER, MerTK TIM-1, TIM-4, TLR3, TLR8, RIG-1, and MDA5. The pharmaceutical composition includes either a purified viral RNA of an oncolytic Zika virus in a liposome and a pharmaceutically acceptable carrier, or a pharmaceutically acceptable carrier and a unit dosage form of an oncolytic Zika virus.

Abstract: A head cradle for supporting a head of a patient during surgery when the patient is in a prone position. The head cradle includes a structural member with a body having a concave profile, a first end and a second end. The body has a forehead portion positioned at the first end for supporting a forehead of a patient and at least one cheek portion for supporting a cheek of the patient. The body includes an aperture defined through the structural member adjacent to the forehead portion between the first end and the second end. The structural member is sized to terminate at a position at or superior to a chin of the patient. A support member is provided to be received by the structural member and to interface with at least a portion of a face of the patient.

Abstract: Skeletonized blood vessels for use as vascular grafts are protected from biomechanical injury and/or certain cellular and extracellular changes by application of a biocompatible hydrogel to the vessel exterior. The hydrogel may be applied to the vessel graft before or after harvesting from a donor patient.

Abstract: An elbow joint boost device has first and second frames to be attached to the forearm and lower arm of a patient. A guide mounted on the first frame extends generally along the respective one of the forearm and upper arm. A link is mounted on the guide for sliding movement along the guide, and a spring acts in tension between the sliding link and a point on the second frame remote from the patient's elbow. As the patient straightens and bends the elbow, the link can slide along the guide, varying the leverage of the tension spring.

Abstract: A chimeric antigen receptor specific for the extracellular membrane-proximal domain (EMPD) of membrane-bound IgE (mIgE) is provided. The EMPD-specific chimeric antigen receptor comprises an extracellular ligand binding domain capable of binding EMPD, a transmembrane domain, and an intracellular domain that mediates T cell activation upon EMPD binding. Nucleic acids and vectors encoding the EMPD-specific chimeric antigen receptor are provided. T cells transduced with such vectors find use in chimeric antigen receptor-based adoptive T-cell therapy for targeting IgE-expressing B cells and treating IgE-mediated allergic diseases.

Abstract: Provided are tumor-activated alloreactive or xenoreactive T cells that are active only at the tumor sites and methods for the generation of tumor-activated alloreactive or xenoreactive T cells. Also provided are methods for using these tumor-activated alloreactive or xenoreactive T cells to treat tumors and cancers. The alloreactivity or xenoreactivity of the T cells at the tumor sites leads to the killing of tumor cells and stromal cells that express mismatched HLA molecules. The lack of activity of these T cells at non-tumor locations prevents attack on normal tissues. Further related methods and products are provided.

Abstract: Objective Pulmonary Function (PF) evaluation for respiratory fatigue is vital to the diagnosis and management of many pediatric respiratory diseases in the intensive care, emergency and outpatient settings. A non-invasive PF instrument utilizes sensors and software to access respiratory breathing patterns, vital parameters, asynchrony and measures the work of breathing. Software algorithms predict respiratory fatigue. The hardware includes a microcircuit board that individually links to rib cage (RC) and abdominal (ABD) inductance bands. The bands wirelessly transmit changes in RC and ABD circumference. Point-of-care, real-time indices of respiratory work, breathing patterns and respiratory fatigue indices are developed on a user-friendly graphical user interface. The diagnostic data can later be securely emailed as an attachment for entry into patients' electronic medical records or sent to a caretaker's computer, or used directly to control a respiratory therapy device.

Abstract: CD24-positive malignant and benign tumors are treated by administration of a naturally occurring or modified oncolytic Zika virus. Diseases associated with abnormal T cell activation or T cell-mediated autoimmunity, wherein CD24 expression is increased, are also expected to be treated by administration of a naturally occurring or modified oncolytic Zika virus. Also contemplated are compounds and methods for treating and/or preventing Zika virus infection in a subject.

Abstract: A therapeutic agent containing, as an effective component, a glycolytic enzyme which is different from a deficient protein of a patient with lysosomal storage disease as a subject and/or a glycolytic enzyme which does not have a mannose 6-phosphate moiety or a mannose moiety.

Abstract: In one aspect, the invention provides a method of detecting cerebral palsy in a subject. The method includes: determining a methylation state for each of a plurality of cytosine-guanine dinucleotide (CpG) sites in a sample obtained from the subject, calculating a differential methylation level based on the methylation states of the plurality of CpG sites, and comparing the differential methylation level to a predetermined reference level. When the differential methylation level deviates from the predetermined reference level, the presence of cerebral palsy is indicated in the patient.

Abstract: Skeletonized blood vessels for use as vascular grafts are protected from biomechanical injury and/or certain cellular and extracellular changes by application of a biocompatible hydrogel to the vessel exterior. The hydrogel may be applied to the vessel graft before or after harvesting from a donor patient.

Abstract: CD24-positive malignant and benign tumors are treated by administration of a naturally occurring or modified oncolytic Zika virus. Diseases associated with abnormal T cell activation or T cell-mediated autoimmunity, wherein CD24 expression is increased, are also expected to be treated by administration of a naturally occurring or modified oncolytic Zika virus. Also contemplated are compounds and methods for treating and/or preventing Zika virus infection in a subject.

Abstract: A chimeric antigen receptor (CAR) contains a human Fc?RI? extracellular domain or a mutant Fc?RI? extracellular domain that has a reduced binding affinity for human IgE compared to wild type human Fc?RI? extracellular domain. The CAR further contains an intracellular signaling domain comprising at least one immunoreceptor-based activation motif (ITAM). The CAR may further comprise a costimulatory signaling domain, such as an intracellular domain of at least one of the costimulatory molecules 4-1BB, CD27, CD28, CD134 or ICOS. T cells transduced with vectors encoding the CAR are used in CAR-based adoptive T-cell therapy for targeting IgE-expressing B cells and treating IgE-mediated allergic diseases.

Abstract: An assay mixture includes a hydrogel of a shear-thinning ?-hairpin peptide, a plurality of cells, and one or more predetermined compounds being investigated for ability to affect the growth, viability, reproduction characteristics, or activity of the cells. A high throughput screening device includes a plurality of sample wells adapted for high throughput screening, wherein each well contains the assay mixture. A method of using the high throughput screening device includes a) depositing in each of the wells a ?-hairpin hydrogel including the cells; depositing in at least some of the wells one or more of the compounds, either along with the ?-hairpin hydrogel or separately; and c) measuring the growth, viability, reproduction characteristics, or activity of the cells in each of the plurality of wells.

Abstract: A retractor for use in lingual frenotomy has a tapered hollow shield, having a closed distal end and an open proximal rim, a groove opening through the distal end to seat over the lingual frenulum, and a handle extending from the proximal rim in line with the groove.

Abstract: Provided is a rapid screening method for detecting mucopolysaccharidosis IVA and mucopolysaccharidosis VII. Biological samples containing glycosaminoglycans are enzymatically digested to converts chondroitin 6-sulfate into ?Di-6S. ?Di-6S is assayed by liquid chromatography-tandem mass spectroscopy analysis. Elevated ?Di-6S, and thus elevated chondroitin 6-sulfate present in the sample, indicates a likelihood that the test subject is afflicted with mucopolysaccharidosis IVA or mucopolysaccharidosis VII.

Abstract: Systems and methods for assisting respiration extrathoracically, particularly useful for augmenting respiration in neonatal patients, including providing a positive pressure to a torso area of a patient. The positive pressure may be delivered to the torso area of the patient while the torso area is exposed to an ambient pressure, such as by providing positive pressure with high frequency gas jets that are positioned in proximity to the torso area. The positive pressure may be delivered to different parts of the torso area of the patient at different times, such as by controlling gas jets independently. The positive pressure may also be controlled in coordination with a gas flow and concentration to the patient's airway, such as by increasing the positive pressure as a gas flow pressure delivered to the patient's airway is reduced.

Abstract: A gravity-balance device, which may be an orthotic support, has a support, a lever pivotally mounted on the support, a spring extending from the support to the lever, and a cam mounted on one of the lever and the support. The cam is positioned so that the spring wraps round the cam as the lever pivots, altering the effective point of action of the spring. A thing being supported can hang from the lever. The thing may be a sling supporting a human arm parallel to the lever, so that the spring compensates for the weight of the arm as the arm is raised and lowered. The cam may be a smooth curve, a polygon, or a row of pins at the corners of an imaginary polygon.